Hematopoiesis News Volume 7.13 | Apr 5 2016

Structure-Activity Relationship Studies of Mitogen Activated Protein Kinase Interacting Kinase (MNK) 1 & 2 and BCR-ABL1 Inhibitors Targeting Chronic Myeloid Leukemic Cells
Clinically used BCR-ABL1 inhibitors for the treatment of chronic myeloid leukemia do not eliminate leukemic stem cells. The authors describe the identification of novel dual MNK1 & 2 and BCR-ABL1 inhibitors, starting from the known kinase inhibitor 2. [J Med Chem] Abstract

Long-Term Restoration of Thymidine Phosphorylase Function and Nucleoside Homeostasis Using Hematopoietic Gene Therapy in a Murine Model of MNGIE
Investigators showed that ectopic expression of thymidine phosphorylase in the hematopoietic system restores normal nucleoside levels in plasma, as well as in tissues affected in mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) such as small intestine, skeletal muscle, brain and liver. [Hum Gene Ther] Abstract

Low C-Kit Expression Level Induced by Stem Cell Factor Does Not Compromise Transplantation of Hematopoietic Stem Cells
Scientists determined whether diminished numbers of c-Kit receptors on hematopoietic stem cells (HSPCs) following their internalization induced by the binding of the cytokine stem cell factor would jeopardize transplantability of HSPCs. [Biol Blood Marrow Transplant] Abstract

Targeting GLI1 Suppresses Cell Growth and Enhances Chemosensitivity in CD34⁺ Enriched Acute Myeloid Leukemia Progenitor Cells
Researchers investigated the role of GLI1, the transcriptional activator of Hh signaling, in acute myeloid leukemia (AML) progenitor cells and explored the anti-AML effects of GLI small-molecule inhibitor GANT61. [Cell Physiol Biochem] Full Article

CLINICAL RESEARCH

Myasthenia Gravis Treated with Autologous Hematopoietic Stem Cell Transplantation
Investigators report seven cases of severe myasthenia gravis treated with autologous hematopoietic stem cell transplant in which consistent, durable, symptom-free, and treatment-free remission was achieved. [JAMA Neurol] Full Article | Press Release

Residual β-Cell Function Predicts Clinical Response after Autologous Hematopoietic Stem Cells Transplantation
Scientists aimed to select the optimal quantitative index to assess pre-existing β-cell function and to explore its predictive function for clinical response after autologous hematopoietic stem cell transplantation therapy. [Stem Cells Transl Med] Abstract

Predictive Value of High EVI1 Expression in AML Patients Undergoing Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation in First CR
Investigators determined whether the overexpression of EVI1 predicts poor outcome of AML patients undergoing myeloablative allogeneic hematopoietic stem cell transplantation in first CR. [Bone Marrow Transplant] Abstract

Arginine Methyltransferases in Normal and Malignant Hematopoiesis
In the hematopoietic system, protein arginine methyltransferases can regulate cell behavior, including the critical balance between stem cell self-renewal and differentiation in at least two critical ways, via the covalent modification of transcription factors, and the regulation of histone modifications at promoters critical to cell fate determination. [Exp Hematol] Abstract | Full Article

Visit our reviews page to see a complete list of reviews in the hematopoiesis research field.

BioLineRx Reports Successful Top-Line Results in Phase II Trial for AML
BioLineRx announced positive top-line results from BL-8040’s Phase II clinical trial in relapsed or refractory acute myeloid leukemia (AML). The BL-8040 oncology platform is a short cyclic peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. [BioLineRx] Press Release

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UC San Diego Health Joins National Clinical Trial on Hemophilia B Gene Therapy
The Hemophilia and Thrombosis Treatment Center at University of California (UC) San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B. [University of California, San Diego] Press Release

SELLAS Life Sciences Group Receives a Favorable Opinion for European Orphan Drug Designations for WT1 Cancer Vaccine (Galinpepimut-S) for the Treatment of Acute Myeloid Leukemia and for Malignant Pleural Mesothelioma
SELLAS Life Sciences Group announced that the Committee for Orphan Medicinal Products of the European Medicines Agency has rendered a favorable opinion as regards Orphan Drug Designation for the Company’s WT1 cancer vaccine (galinpepimut-S) for the treatment of patients with acute myeloid leukemia and patients with malignant pleural mesothelioma. [SELLAS Life Sciences Group] Press Release

American Cancer Society Awards New Research and Training Grants
The American Cancer Society has approved funding for 103 research and training grants totaling nearly $44 million in the first of two grant cycles for 2016. The grants will fund investigators at 74 institutions across the United States; 95 are new grants while eight are renewals of previous grants. [The American Cancer Society] Press Release

Food and Drug Administration (United States)

Center for Biologics Evaluation and Research (United States)

European Medicines Agency (European Union)

Medicines and Healthcare Products Regulatory Agency (United Kingdom)

Therapeutic Goods Administration (Australia)

Visit our events page to see a complete list of events in the hematopoiesis community.

Scientist – Hematology (STEMCELL Technologies Inc.)

Postdoctoral Scientist – Hematological Malignancies (Max Delbruck Center for Molecular Medicine in the Helmholtz Association)

Postdoctoral Fellow – Molecular and Cellular Basis of Hematopoiesis (University of Cincinnati)

Postdoctoral Research Associate – Blood Stem cell Microenvironment (University of Illinois)

Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

Postdoctoral Fellow Positions – Leukemogenesis (The University of Alabama)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Fellow – Cancer Stem Cell Biology (McGill University Health Center)

Postdoctoral Fellow – Cancer Biology (National University of Singapore)

Assistant/Associate/Full Member Faculty – Myeloid Malignancies (Fred Hutchinson Cancer Research Center)

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